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New hope for kids with rare copper deficiency disease

NCT ID NCT04074512

Disease control Sponsor: Sentynl Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved This study
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This program gives children under 6 with Menkes disease access to copper histidinate injections. Menkes disease is a rare genetic disorder where the body cannot properly use copper, leading to severe developmental problems. The treatment aims to supply the missing copper to help manage the condition.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
copper histidinate (CUTX-101)
What this could lead to
If this treatment works, it could help manage Menkes disease by providing the copper the body needs, potentially improving survival and development in young children.
What could go wrong
This is an expanded access program, not a controlled trial, so results are not definitive. The treatment may not work for all patients, and side effects from copper therapy are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 6 years

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. The subject must have been treated in protocol 09-CH-0059 or must be a newly diagnosed Menkes disease patient in the US. 2. Parent or legal guardian is willing and able to sign and date an informed consent form. 3. Male or female, aged 0 to \<6 years of age. 4. Diagnosis of Menkes disease: A confirmed gene mutation in ATP7A is not required; however, the test must be at least pending prior to initiating therapy. For those patients whose molecular ATP7A gene mutation confirmation is pending, they should have serum copper \<75 mcg/dL. If the mutation is subsequently not confirmed, the patient should discontinue treatment. 5. Ability to adhere to the prescribed subcutaneous copper histidinate injection regimen. 6. Willingness to adhere to all recommended visits and procedures. 7. Resident of the US. Exclusion Criteria 1. Diagnosis of Wilson disease. 2. Any disease or condition that, in the opinion of the Investigator, has a high probability of precluding the subject from completing the study or where the subject cannot or will not appropriately comply with study requirements. 3. Receipt of concomitant medication or device not approved for any use (i.e. experimental) by FDA within 30 days prior to screening for this study, except for participation in protocol 09-CH-0059. 4. Patient/caregiver cannot or will not appropriately comply with protocol recommendations (e.g., site visits, renal monitoring). 5. Currently receiving any concomitant copper-containing medications.

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Conditions

The condition(s) this trial relates to.

Menkes disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Menkes disease

Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106, United States

  • Cook Children's Health Care System

    Fort Worth, Texas, 76104, United States

  • Duke University Hospital

    Durham, North Carolina, 27710, United States

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503, United States

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701, United States

  • Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Maine Medical Center

    Portland, Maine, 04102, United States

  • Memorial Healthcare Systems

    Hollywood, Florida, 33021, United States

  • Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Shodair Children's Hospital

    Helena, Montana, 59601, United States

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa, 52242, United States

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